Skip to main content
Publications
Desai RJ, Wang SV, Sreedhara SK, Zabotka L, Khosrow-Khavar F, Nelson JC, Shi X, Toh S, Wyss R, Patorno E, Dutcher S, Li J, Lee H, Ball R, Dal Pan G, Segal JB, Suissa S, Rothman KJ, Greenland S, Hernan MA, Heagerty PJ, Schneeweiss S. Process guide for inferential studies using healthcare data from routine clinical practice to evaluate causal effects of drugs (PRINCIPLED): considerations from the FDA Sentinel Innovation Center. BMJ. 2024 Feb 12;384:e076460. doi: 10.1136/bmj-2023-076460
Forsythe E, Haws RM, Argente J, Beales P, Martos-Moreno GA, Dollfus H, Chirila C, Gnanasakthy A, Buckley BC, Mallya UG, Clement K, Haqq AM. Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. Orphanet J Rare Dis. 2023 Jan 16;18(1):12. doi: 10.1186/s13023-022-02602-4
Kuhnen P, Wabitsch M, von Schnurbein J, Chirila C, Mallya UG, Callahan P, Gnanasakthy A, Poitou C, Krabusch PM, Stewart M, Clement K. Quality of life outcomes in two phase 3 trials of setmelanotide in patients with obesity due to LEPR or POMC deficiency. Orphanet J Rare Dis. 2022 Feb 5;17(1):38. doi: 10.1186/s13023-022-02186-z
Ackley SF, Zimmerman SC, Brenowitz WD, Tchetgen Tchetgen EJ, Gold AL, Manly JJ, Mayeda ER, Filshtein TJ, Power MC, Elahi FM, Brickman AM, Glymour MM. Effect of reductions in amyloid levels on cognitive change in randomized trials: instrumental variable meta-analysis. BMJ. 2021 Feb 25;372:n156. doi: 10.1136/bmj.n156
Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien Ansquer V, Germain DP, Lidove O. Treatment needs and expectations for Fabry disease in France: development of a new Patient Needs Questionnaire. Orphanet J Rare Dis. 2019 Dec 4;14(1):284. doi: 10.1186/s13023-019-1254-7
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay H. Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best–worst scaling experiment in caregivers and adult patients. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6
Obel N, Dessau RB, Krogfelt KA, Bodilsen J, Andersen NS, Moller JK, Roed C, Omland LH, Christiansen CB, Ellermann-Eriksen S, Bangsborg JM, Hansen K, Benfield TL, Rothman KJ, Sorensen HT, Andersen NS, Lebech AM. Long term survival, health, social functioning, and education in patients with European Lyme neuroborreliosis: nationwide population based cohort study. BMJ. 2018 May 30;361:k1998. doi: 10.1136/bmj.k1998
Henry D, Lim LL, Garcia Rodriguez LA, Perez GS, Carson JL, Griffin M, Savage R, Logan R, Moride Y, Hawkey C, Hill S, Fries JT. Variability in risk of gastrointestinal complications with individual non-steroidal anti-inflammatory drugs: results of a collaborative meta-analysis. BMJ. 1996 Jun 22;312(7046):1563-6.